Amicus gets hit with an ‘unexpected’ setback on a gene therapy tapped to lead $600M SPAC spinout

John Crowley, Amicus CEO (via YouTube)

January 11, 2022 06:49 AM ESTUpdated 07:00 AM

Amicus gets hit with an 'unexpected' setback on a gene therapy tapped to lead $600M SPAC spinout

Amicus has taken the axe and chopped a gene therapy for Batten disease out of the pipeline after an “unexpected” setback in the clinic.

In a corporate update on Monday, the biotech reported that its long-term extension data from a Phase I/II study revealed that the initial stabilization they had achieved in patients during the first 2 years wasn’t sustainable. So researchers are dropping the CLN6 Batten disease gene therapy program and turning to a CLN3 program, also for Batten disease, with a “higher dose, different promoter, and intra-cisterna magna route of delivery.”

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Tom Avery

Scientific and Medical Affairs Specialist, Medical Devices

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August 13, 2022 06:00 AM EDT

Weekly

Historic drug pricing reforms pass; Pfizer acquires GBT; The long search for non-opioid pain drugs; and more

Amber Tong

Senior Editor

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

The Endpoints Weekly has officially crossed the 60,000 mark on subscribers — thanks to all of your support. As the editorial team grows, we’ve been able to do a lot more, with many of those on display this week. Be sure to check out Lei Lei Wu’s deep dive on pain R&D. If you missed it, you may also rewatch her companion panel here.

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August 12, 2022 09:19 AM EDT

R&D

Pharma

Gold for adults, silver for infants: Pfizer's Prevnar 2.0 headed to FDA months after Merck's green light

Kyle LaHucik

Associate Editor

Pfizer was first to the finish line for the next-gen pneumococcal vaccine in adults, but Merck beat its rival with a jab for children in June.

Now, two months after Merck’s 15-valent Vaxneuvance won the FDA stamp of approval for kids, Pfizer is out with some late-stage data on its 20-valent shot for infants.

Known as Prevnar 20 for adults, Pfizer’s 20vPnC will head to the FDA by the end of this year for an approval request in infants, the Big Pharma said Friday morning. Discussions with the FDA will occur first and more late-stage pediatric trials are expected to read out soon, informing the regulatory pathway in other countries and regions.

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ENDPOINTS CAREERS

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Experimental Drug Development Centre

Singapore

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August 12, 2022 07:22 AM EDT

Pharma

Novartis reports two patient deaths after treatment with Zolgensma

Amber Tong

Senior Editor

Two children with spinal muscular atrophy have died after receiving Novartis’ Zolgensma, a gene therapy designed as a one-time treatment for the rare fatal disease.

The deaths, which resulted from acute liver failure, occurred in Russia and Kazakhstan, Novartis confirmed in a statement to Endpoints News. Having notified health authorities across all the markets where Zolgensma is available, it will update the drug label “to specify that fatal acute liver failure has been reported,” a spokesperson wrote.

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Benjamin Oakes, Scribe Therapeutics CEO

August 12, 2022 07:46 AM EDTUpdated 01:01 PM

Cell/Gene Tx

CEO of Doudna spinout: Within five years, genome editors will have a 'really big impact' on patients' lives

Zachary Brennan

Senior Editor

“CRISPR-by-design” is the idea behind Scribe Therapeutics, a company spun out from Jennifer Doudna’s Nobel-winning lab that’s competing in a closely-tracked field of genome editor companies just starting to make their way to the clinic.

After nabbing $100 million last March for its Series B funding round, Scribe is taking a different tack from some of its competitors, crafting a new enzyme isolated from bacteria called CasX, which has now been tweaked extensively and may be targeted to a range of genome-related diseases, offering a plethora of therapeutic options.

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August 12, 2022 05:36 PM EDTUpdated 4 minutes ago

Law

House passes historic drug pricing reforms, lining up decades-in-the-making win for Biden and Democrats

Zachary Brennan

Senior Editor

The US House of Representatives today voted along party lines (all Dems voted for it), 220-207 to pass new, wide-ranging legislation that will allow Medicare drug price negotiations for the first time ever, and cap seniors’ drug expenses to $2,000 per year and seniors’ insulin costs at $35 per month.

Setting up a major victory for President Joe Biden, representatives returned from their summer recess to pass the Inflation Reduction Act, even as many noted the bill would only modestly reduce inflation.

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Senate Finance Committee Chair Ron Wyden (D-OR) (Francis Chung/E&E News/POLITICO via AP Images)

August 12, 2022 02:02 PM EDTUpdated 03:16 PM

Pharma

Law

Senate Finance chair continues his investigation into pharma taxes with requests for Amgen

Zachary Brennan

Senior Editor

Amgen is the latest pharma company to appear on the radar of Senate Finance Committee Chair Ron Wyden (D-OR), who is investigating the way pharma companies are using subsidiaries in low- or zero-tax countries to lower their tax bills.

Like its peers Merck, AbbVie and Bristol Myers Squibb, Wyden notes how Amgen uses its Puerto Rico operations to consistently pay tax rates that are substantially lower than the U.S. corporate tax rate of 21%, with an effective tax rate of 10.7% in 2020 and 12.1% in 2021.

ENDPOINTS CAREERS

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August 12, 2022 12:47 PM EDTUpdated 02:10 PM

Pharma

FDA+

FDA approves second indication for AstraZeneca and Daiichi's Enhertu in less than a week

Aayushi Pratap

News Reporter

AstraZeneca and Daiichi Sankyo’s antibody-drug conjugate Enhertu scored its second approval in less than a week, this time for a subset of lung cancer patients.

Enhertu received accelerated approval on Thursday to treat adults with unresectable or metastatic non-small cell lung cancer (NSCLC) whose tumors have activating HER2 (ERBB2) mutations, and who have already received a prior systemic therapy.

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August 12, 2022 12:39 PM EDT

Pharma

J&J to remove talc products from shelves worldwide, replacing with cornstarch-based portfolio

Nicole DeFeudis

Editor

After controversially spinning out its talc liabilities and filing for bankruptcy in an attempt to settle 38,000 lawsuits, Johnson & Johnson is now changing up the formula for its baby powder products.

J&J is beginning the transition to an all cornstarch-based baby powder portfolio, the pharma giant announced on Thursday — just months after a federal judge ruled in favor of its “Texas two-step” bankruptcy to settle allegations that its talc products contained asbestos and caused cancer. An appeals court has since agreed to revisit that case.